June 28 2013 | By Márcio Barra
The US Food and Drug Administration granted yesterday U.S. marketing approval for Rixubis, a genetically engineered protein (Coagulation Factor IX (Recombinant)) for the treatment of hemophilia B, made by Baxter International Inc. Hemophilia is an inherited blood clotting disorder that mainly affects males. Hemophilia B is one of two forms of the disease, and is caused by mutations in the Factor IX gene and leads to deficiency of Factor IX, causing excessive bleeding. The most common form is hemophilia A.
This is the first new treatment for Hemophilia in more than 15 years.
Rixubis is administered intravenously twice weekly to control and prevent bleeding in hemophilia B patients in the hospital for surgery. It can also be used routinely to prevent or reduce bleeding.
“As the first recombinant coagulation factor IX indicated specifically for routine prophylaxis to prevent bleeding, Rixubis becomes a new weapon in our arsenal to protect Hemophilia B patients,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.
FDA’s approval was based on a late-stage trial with73 male patients between 12 and 65 years of age. Overall, patients in the prophylaxis study had a 75 percent lower annual bleeding rate when compared to patients who were treated only when bleeding episodes occurred. Most common side effects included distorted taste, extreme pain, atypical blood test results and anaphylaxis, a life-threatening allergic reaction. Baxter now plans to study Rixubis in the pediatric population.
Baxter expects to file for marketing authorization for RIXUBIS in the European Union later this year.